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Pediatric Rhabdomyosarcoma Treatment & Management

  • Author: Timothy P Cripe, MD, PhD, FAAP; Chief Editor: Max J Coppes, MD, PhD, MBA  more...
Updated: May 01, 2016

Medical Care

Treatment in patients with rhabdomyosarcoma (RMS) involves a combination of surgery, chemotherapy, and radiation therapy. Because the treatment plan is complicated and prolonged and because many medical issues are unique to pediatric oncology, all patients should be referred (at least initially) to a center with personnel who are skilled in caring for children with cancer.

At present, patients are categorized according to their risk, which takes into account the location of the tumor and the histologic and surgical results. Low-risk patients are those who have the best prognosis, whereas intermediate-risk or high-risk patients have an increased risk of having relapses and incurable disease. To separate the features into meaningful categories, patients are assigned to both a surgicopathologic clinical group (Roman numeral) and a stage (Arabic numeral). All patients with metastatic disease (group IV, stage 4) are considered high risk, except children and adolescents younger than 14 years with embryonal rhabdomyosarcoma (ERMS). In some studies, these patients appear to do better than others, for unknown reasons. Although all patients require chemotherapy, regimens vary depending on the stage and group.

  • Surgicopathologic (clinical) group (Groups I-III are for localized disease).
    • Group I - Tumor completely removed
    • Group II - Microscopic residual tumor, involved regional nodes, or both
    • Group III - Gross residual tumor
    • Group IV - Distant metastatic disease
  • Tumor, nodes, and metastases (TNM) staging system
    • Tumor - Confined to the site of origin (T1) or extends beyond the site of origin (T2)
    • Node - No regional node involvement (N0), regional node involvement (N1), or nodes unknown (NX)
    • Metastasis - No metastasis (M0), or metastases present at diagnosis (M1)
  • RMS staging system
    • Stage 1 - Orbit, head, and/or neck (not parameningeal) involvement, and involvement of the GU tract (not bladder or prostate)
    • Stage 2 - Other locations, N0 or NX
    • Stage 3 - Other locations, N1 if the tumor is less than 5 cm or N0 or NX if the tumor more than 5 cm
    • Stage 4 - Any site with distant metastases
  • Low-risk patients are those with the following embryonal histology:
    • Stages 1-3 in groups I-II (or III for only orbital involvement)
    • Stage 1 in group III

A study by Weigel et al that enrolled 109 patients, mostly 20 years of age or younger, with stage IV (metastatic) rhabdomyosarcoma, looked to improve outcome of patients with metastatic rhabdomyosarcoma by dose intensification with interval compression, use of the most active agents determined in phase II window studies, and use of irinotecan as a radiation sensitizer. The study reported a 3-year event-free survival (EFS) rate of 38%, and an overall survival rate of 56% which was below the primary objective to improve the 3-year EFS to 55%. Despite those setbacks, the study did find that patients with metastatic rhabdomyosarcoma with one or no Oberlin risk factor had an improved 3-year EFS of 69% on ARST0431 compared with a historical cohort from pooled European and US studies.[20, 21]


Surgical Care

Surgical management of rhabdomyosarcoma varies depending on the location of the tumor. If feasible, remove tumors promptly and without unacceptable disfigurement or loss of function. Even if metastatic disease is present, surgical excision of the primary site should be performed, if possible. The surgical result helps determine the clinical grouping to be used for treatment stratification.

Surgical guidelines for the various sites can be found in the protocols of the Children's Oncology Group Soft Tissue Sarcoma Committee (formerly, Intergroup Rhabdomyosarcoma Study Group [IRSG]) and are beyond the scope of this article. However, common principles are noteworthy and described below.

  • Primary tumor
    • Because relapses often occur at the site of the original primary tumor, adequate local control is essential.
    • Data from Europe suggest that chemotherapy alone can be effective for achieving adequate local control in some patients who have a complete response of the primary tumor. However, surgery and/or irradiation are needed for local control of residual disease.
    • If possible, complete excision of the lesion should be performed with a wide (2-cm) margin of healthy tissue. Wide margins of normal tissue often are impossible to achieve at certain sites, such as the head and neck. If margins are narrow, obtain several biopsy specimens from the surrounding tissue to assess for residual local disease.
    • For tumors that cannot be excised at diagnosis, a second-look procedure may be appropriate after a period of chemotherapy (usually 12 wk).
  • Lymph nodes
    • Regional lymph nodes that appear to be clinically or radiographically involved should be sampled to determine the clinical group and the need for later radiation therapy.
    • Radical node dissection is not appropriate.
    • Axillary and femoral node sampling should be performed for lesions in the extremities, even if clinical findings are negative because of the high prevalence of metastatic disease arising from extremity lesions.
    • The use of sentinel node biopsy instead of lymph node sampling is being explored.[22]


The care of patients with rhabdomyosarcoma is complicated and extensive and touches all aspects of their lives. Initial evaluation and treatment should be undertaken at a center with a comprehensive program for children with cancer.

  • Radiotherapist
    • Most patients with rhabdomyosarcoma require radiotherapy to achieve adequate local control, though radiotherapy is not usually performed until after initial surgical resection and the start of chemotherapy. Exceptions are patients with parameningeal primary tumors, for which initial radiotherapy has been shown to be beneficial.
    • A radiotherapist familiar with the requirements for clinical trials should be consulted at diagnosis for most patients in North America to determine if they have any special needs for treatment planning.
    • In initial studies, new techniques such as intensity-modulated radiotherapy (IMRT) and proton-beam radiotherapy appear to achieve adequate tumor control with reduced exposure to normal tissues.[23, 24]
    • There is a high risk of local recurrence in patients with group II disease (microscopic residual disease) if prescribed radiotherapy is omitted or reduced, as demonstrated by Million et al.[25] They noted that more than half the patients who relapsed at the original tumor site received nonstandard radiation therapy, and, of these, three quarters died of their disease.
  • Psychosocial team: The psychosocial team is critical for helping patients and families cope with the stresses associated with the diagnosis and treatment of cancer. The social worker usually plays an intimate role in helping families navigate complicated insurance and financial issues.
  • Dentist: A thorough dental evaluation is required to identify potential problems that may arise during chemotherapy.
  • Pediatric therapists: Activities and therapy can be critical in helping the patient through the phases of medical therapy.


No specific dietary recommendations are needed. However, patients may require nasogastric feedings or parenteral nutrition during some phases of chemotherapy. This is especially true for patients with primary tumors in head and neck, who may have severe mucositis after radiation therapy.



No specific activity limitations are required. The patient's activity is restricted only as the location of the tumor and the adverse effects of treatment dictate.

Contributor Information and Disclosures

Timothy P Cripe, MD, PhD, FAAP Chief, Division of Hematology/Oncology/BMT, Gordon Teter Endowed Chair in Pediatric Cancer, Nationwide Children's Hospital; Professor of Pediatrics, Ohio State University College of Medicine

Timothy P Cripe, MD, PhD, FAAP is a member of the following medical societies: American Academy of Pediatrics, American Association for the Advancement of Science, American Association for Cancer Research, American Pediatric Society, American Society of Gene and Cell Therapy, American Society of Pediatric Hematology/Oncology, Connective Tissue Oncology Society, Society for Pediatric Research, Children's Oncology Group

Disclosure: Nothing to disclose.

Specialty Editor Board

Mary L Windle, PharmD Adjunct Associate Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Nothing to disclose.

Steven K Bergstrom, MD Department of Pediatrics, Division of Hematology-Oncology, Kaiser Permanente Medical Center of Oakland

Steven K Bergstrom, MD is a member of the following medical societies: Alpha Omega Alpha, Children's Oncology Group, American Society of Clinical Oncology, International Society for Experimental Hematology, American Society of Hematology, American Society of Pediatric Hematology/Oncology

Disclosure: Nothing to disclose.

Chief Editor

Max J Coppes, MD, PhD, MBA Executive Vice President, Chief Medical and Academic Officer, Renown Heath

Max J Coppes, MD, PhD, MBA is a member of the following medical societies: American College of Healthcare Executives, American Society of Pediatric Hematology/Oncology, Society for Pediatric Research

Disclosure: Nothing to disclose.


Samuel Gross, MD Professor Emeritus, Department of Pediatrics, University of Florida College of Medicine; Clinical Professor, Department of Pediatrics, University of North Carolina at Chapel Hill School of Medicine; Adjunct Professor, Department of Pediatrics, Duke University School of Medicine

Samuel Gross, MD is a member of the following medical societies: American Association for Cancer Research, American Society for Blood and Marrow Transplantation, American Society of Clinical Oncology, American Society of Hematology, and Society for Pediatric Research

Disclosure: Nothing to disclose.

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Axial CT scan of rhabdomyosarcoma in the left middle ear. Image provided by Suresh Muhkerji, MD, Department of Radiology, University of North Carolina Hospitals.
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